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Biomedical subjects

C L Bennett

Publications and source records attributed to C L Bennett.

At least 91 records · Page 5Linked to original sources

Pharmacoeconomics of amifostine in ovarian cancer.

Physicians are frequently pressured to make therapeutic decisions within a cost-effective framework to demonstrate value to managed care. Because cancer is a chronic disease, health care costs are known to be expensive and physicians must use their resources as efficiently as possible. Historically, economic analyses in oncology have emphasized survival as their clinical end point. Today, both government groups and professional organizations are moving toward making quality of life the clinical end point in determining the economics of chemotherapy. This report evaluates the cost and efficacy of amifostine (Ethyol; Alza Pharmaceuticals, Palo Alto, CA/US Bioscience, West Conshohocken, PA) use in the treatment of advanced ovarian cancer using two pharmacoeconomic analyses. A cost-utility analysis performed in the United States indicated that inclusion of amifostine therapy had both a favorable clinical and cost-utility profile compared with other medical therapies. A second cost-benefit analysis, conducted in Canada, suggested that use of amifostine in patients with advanced ovarian cancer would be cost saving. Amifostine is a novel agent that protects against both chemotherapy- and radiotherapy-induced toxicities, such as nephrotoxicity, neutropenia, thrombocytopenia, peripheral neuropathy, mucositis, and xerostomia. These toxicities are disturbing to both patients and physicians alike. The results of these studies support the use of amifostine as a valuable resource both economically and clinically.

Amifostine↗

QOL and outcomes research in prostate cancer patients with low socioeconomic status.

The VA Cancer of the Prostate Outcomes Study (VA CaPOS) is collecting quality-of-life (QOL) information from prostate cancer patients, spouses, and physicians at six VA medical centers. Currently, 601 men with prostate cancer are included in the study, most of whom are of low socioeconomic status and over half of whom are African-American. Quality-of-life responses were most favorable for newly diagnosed patients, intermediate for those with stable metastatic disease, and poorest for those with progressive metastatic disease. Patients could not provide reliable estimates of their own preferences for future QOL states but responded reliably to questions phrased as a comparison of the preferences of two hypothetical patients. High out-of-pocket costs for hormonal therapies, lack of health insurance, and a belief that the non-VA system offered poorer services were the most common reasons for patient transferral to the VA system. Satisfaction with medical care was generally high. While African-American patients were more likely to have advanced prostate cancer at diagnosis, after adjustment for differences in health literacy, race was no longer a significant predictor of advanced disease. The VA CaPOS provides useful information on health status and patient satisfaction of VA prostate cancer patients. Long-term evaluations are needed to detect clinically meaningful QOL information as the disease progresses.

Adult↗

Cost-effectiveness model of a phase II clinical trial of a new pharmaceutical for essential thrombocythemia: is it helpful to policy makers?

Essential thrombocythemia (ET) is a rare, chronic myeloproliferative disorder of unknown origin characterized by thrombocytosis, excessive megakaryocytes, hemorrhages, and thrombotic complications. Because of the high costs of care for persons with rare diseases, policymakers are concerned with both clinical effectiveness and cost-effectiveness of new treatments. Although the clinical efficacy of all new pharmaceutical agents for rare diseases is evaluated extensively in clinical trial settings before approval by the Food and Drug Administration (FDA), comparative phase III trials of a new agent with its major competitor are sometimes not possible to carry out, and estimates of cost-effectiveness are therefore difficult to obtain. We describe methodologic issues associated with the development of economic models of new pharmaceutical agents for rare diseases and illustrate these issues with an analysis of a new therapy for ET. Anagrelide is a newly approved platelet aggregation inhibitor that can be used as primary therapy for ET. The agent reduces platelet counts by 50% in more than 70% of ET patients. Economic models suggest that, over the first year of anagrelide therapy, monthly costs for therapy and complications decreased from $775 to $490, the effectiveness improved to 98%, and the cost-effectiveness improved to $1,505 per major complication (gastrointestinal bleed, transient ischemic attack or stroke, or preinfarction angina or myocardial infarction) prevented. Sensitivity analyses indicate that, after the first 3 months of treatment, total costs of anagrelide treatment were in the range of $1,505 to $1,615 per major complication prevented. To make well-informed therapeutic decisions, policymakers and physicians require head-to-head studies of a new pharmaceutical agent with its major competitor. However, economic models can be used to derive estimates of cost-effectiveness of new pharmaceutical agents when such data are lacking. The interpretation of these models raises general issues related to the perspective of the investigator, study design, estimation of costs of care, rates of response, toxicity, survival, and the ability to generalize the results to other settings, as well as methodologic issues that are unique to rare diseases. If a comparative study found better therapeutic outcomes, then cost-effectiveness models would be of limited usefulness. Almost all physicians would use the drug with the better therapeutic profile.

Clinical Trials, Phase II as Topic↗

Cost-effectiveness analysis comparing liposomal anthracyclines in the treatment of AIDS-related Kaposi's sarcoma.

Liposomal formulations have been shown to alter the efficacy and toxicity profiles of anthracylines for patients with HIV-related advanced Kaposi's sarcoma (KS). Using decision-analysis models, the costs and cost-effectiveness of the two U.S. Food and Drug Administration (FDA)-approved liposomal formulations of these agents were estimated. Estimates of costs, effectiveness, and cost-effectiveness were derived from clinical trial data of separate, randomized phase III trials of pegylated liposomal doxorubicin (20 mg/m2 every 3 weeks) and liposomal daunorubicin (40 mg/m2 every 2 weeks). Clinical response rates were 59% for pegylated liposomal doxorubicin and 25% for liposomal daunorubicin. Despite higher acquisition costs for pegylated liposomal doxorubicin, total estimated costs of treatment for KS and chemotherapy-related hematologic toxicities were similar ($7,066 U.S. compared with $6,621 U.S. for liposomal daunorubicin). Cost-effectiveness profiles, defined as average costs per responder, favored pegylated liposomal doxorubicin ($11,976 U.S./responder versus $26,483 U.S./responder for liposomal daunorubicin), reflecting the higher reported response rate in the phase III trial. Sensitivity analyses suggested that the costs and cost-effectiveness results would not differ markedly when evaluated over a range of assumptions, including response rate, neutropenia rate, and dosage variations.

Acquired Immunodeficiency Syndrome↗

Thrombotic thrombocytopenic purpura associated with ticlopidine. A review of 60 cases.

BACKGROUND: Thrombotic thrombocytopenic purpura, a life-threatening multisystem disease, has been infrequently associated with use of ticlopidine, a platelet anti-aggregating agent. PURPOSE: To review 60 cases of ticlopidine-associated thrombotic thrombocytopenic purpura. DATA SOURCES: Medical records, published case reports, and case reports submitted to the U.S. Food and Drug Administration. STUDY SELECTION: Instances of ticlopidine-associated thrombotic thrombocytopenic purpura were identified. DATA SYNTHESIS: Ticlopidine had been prescribed for less than 1 month in 80% of the patients, and normal platelet counts had been found within 2 weeks of the onset of thrombotic thrombocytopenic purpura in most patients. Mortality rates were higher among patients who were not treated with plasmapheresis than among those who underwent plasmapheresis (50% compared with 24%; P < 0.05). CONCLUSIONS: Ticlopidine use may be associated with the development of thrombotic thrombocytopenic purpura, usually within 1 month of initiation of therapy. The onset of ticlopidine-associated thrombotic thrombocytopenic purpura is difficult to predict, despite close monitoring of platelet counts.

Adult↗

Perceptions of cisplatin-related toxicity among ovarian cancer patients and gynecologic oncologists.

BACKGROUND: We conducted a pilot study to evaluate issues related to chemotherapy-induced toxicities by eliciting assessments of toxicity from women with advanced stage ovarian cancer and gynecologic oncologists. PATIENTS AND METHODS: Fifteen ovarian cancer patients and ten gynecologic oncologists completed the survey exercises. All patients surveyed had received at least six courses of a cisplatin-containing chemotherapy regimen. RESULTS: For both patients and physicians, there was good face validity to the utility exercise as assessments of health states with cisplatin were (1) consistently associated with less favorable assessments than the health state with no toxicity and (2) neurotoxicity was viewed less favorably than either ototoxicity or nephrotoxicity. While the 15 patients as a group viewed health states with toxicity more favorably than physicians (P < 0.05 for each toxicity), patient assessments varied, depending on individual experiences with cisplatin. Physician assessments of toxicity were most similar to those obtained from patients who had not experienced cisplatin toxicity and were less favorable than those elicited from patients who had experienced any toxicity. CONCLUSIONS: In deciding upon therapeutic strategies, women with advanced stage ovarian cancer and treating physicians markedly differ in their assessment of the impact of specific toxicities on quality of life.

Adult↗

Performance measurement in pneumonia care: beyond report cards.

OBJECTIVE: To compare the medical management of bacteremic pneumococcal pneumonia at a university-based and a community-based teaching hospital and evaluate strategies for performance measurement and subsequent improvement. DESIGN: We conducted a retrospective cohort study involving a 450-bed university hospital in the inner city and a 400-bed private hospital in a rural community. MATERIAL AND METHODS: The medical records of all adults with bacteremic pneumococcal pneumonia admitted to a university and a community hospital during a 5-year period were reviewed. Information about patient age, sex, underlying medical condition, severity of disease, health-care insurance, management, and outcome was collected and analyzed. RESULTS: Patients at the two hospitals were similar in underlying illnesses and severity of disease. In comparison with the community hospital, resource expenditure was greater at the university hospital, where all 11 identified diagnostic measures and treatment resources were used more often. This difference was statistically significant for sputum cultures, all cultures, and lumbar punctures. Despite the greater intensity of care, in-hospital mortality was higher at the university hospital (26%) than at the community hospital (12%) (P>0.1). CONCLUSION: The outcome of bacteremic pneumococcal pneumonia did not differ significantly at a university hospital in comparison with a community teaching hospital, even though resource expenditure at the university hospital was greater. Our findings suggest that hospital "report cards" based solely on outcome comparisons provide inadequate information. In contrast, examination of variations in profiles of resource utilization can detect important differences in hospitals and can be used to guide continuous quality improvement efforts and ultimately improve hospital care.

APACHE↗

Economic analyses of bone marrow and blood stem cell transplantation for leukemias and lymphoma: what do we know?

The use of blood and/or bone marrow stem cell transplantation (SCT) grew extensively in the last decade as technological advances led to improved outcomes and wider availability. The first study of SCT costs, however, was not published until 1989. This paper summarizes current knowledge about costs and cost-effectiveness of allogeneic and autologous SCT for leukemias and lymphoma. Methodological issues in cost studies such as types of costs, methods of data collection, and time horizons are discussed, and studies are evaluated with regard to these issues. Considerations specific to economic analyses of SCT are considered, including the potential impact of technological changes, learning curve effects, and inter-institutional differences.

Bone Marrow Transplantation↗

Variations in intensive care unit utilization for patients with human immunodeficiency virus-related Pneumocystis carinii pneumonia: importance of hospital characteristics and geographic location.

OBJECTIVE: To determine whether intensive care unit (ICU) use and outcomes for patients with human immunodeficiency virus (HIV)-related Pneumocystis carinii pneumonia vary by hospital characteristics and geographic location. DESIGN: Retrospective review of the medical records of 2,174 patients with HIV-related P. carinii pneumonia. SETTING: Random sample of 73 private, nine public, and 14 Veterans Affairs hospitals in five cities (Chicago, New York, Los Angeles, Miami, and Durham, NC). PATIENTS: Stratified random sample of patients hospitalized with HIV-related P. carinii pneumonia from 1987 to 1990. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: Among the 2,174 patients with P. carinii pneumonia, 398 (18%) patients received care in an ICU. ICU utilization varied significantly by patient and hospital characteristics, as well by as geographic location. Non-Hispanic whites, patients with Medicaid, and patients with a prior acquired immunodeficiency syndrome-defining illness were the least likely to receive care in an ICU. Patients in county- or state-owned hospitals and patients in hospitals with more P. carinii pneumonia-experience were also less likely to be cared for in an ICU. These differences in ICU utilization persisted when controlling for severity of illness, as well as other patient characteristics. Significant geographic variation in ICU utilization persisted after controlling for patient and hospital characteristics. Survival to hospital discharge after an ICU stay was significantly higher for patients without a prior acquired immunodeficiency syndrome-defining illness and for patients in hospitals with more P. carinii pneumonia experience. CONCLUSIONS: We found significant variations in ICU utilization by hospital characteristics and geographic location that remained significant after controlling for severity of illness and patient sociodemographic characteristics. Hospital and geographic variations in ICU utilization may make it difficult to generalize ICU outcomes across different hospitals.

AIDS-Related Opportunistic Infections↗

Assessing the impact of continuous quality improvement on clinical practice: what it will take to accelerate progress.

The literature on continuous quality improvement (CQI) has produced some evidence, based on nonrandomized studies, that its clinical application can improve outcomes of care while reducing costs. Its effectiveness is enhanced by a nucleus of physician involvement, individual practitioner feedback, and a supportive organizational culture. The few randomized studies, however, suggest no impact of CQI on clinical outcomes and no evidence to date of organization-wide improvement in clinical performance. Further, most studies address misuse issues and avoid examining overuse or underuse of services. The clinical application of CQI is more likely to have a pervasive impact when it takes place within a supportive regulatory and competitive environment, when it is aligned with financial incentives, and when it is under the direction of an organizational leadership that is committed to integrating all aspects of the work.

Ambulatory Care↗

Relation between literacy, race, and stage of presentation among low-income patients with prostate cancer.

PURPOSE: Diagnosis of advanced prostate cancer is a major health problem, especially among low-income men. Opportunities vary for early detection of prostate cancer for low-income black and white men because of financial, cultural, and social factors. In this study, we evaluated the association of poor literacy skills with higher rates of presentation of advanced stages of prostate cancer among low-income black and white men who received care in equal-access medical systems. PATIENTS AND METHODS: Literacy and stage at diagnosis of prostate cancer were evaluated in 212 low-income men who received medical care in Shreveport, LA, and Chicago, IL. The patients' literacy was assessed with the Rapid Estimate of Adult Literacy in Medicine (REALM), an individually administered reading screening test designed specifically for use in the medical setting. Logistic regression models were used to evaluate predictors of metastatic disease at presentation as a function of patient age, race, literacy, and city. RESULTS: Whereas black men were almost twice as likely to present with stage D prostate cancer (49.5% v 35.9%; P < .05), they were significantly more likely to have literacy levels less than sixth grade (52.3% v 8.7%; P < .001). However, after adjustment for differences in literacy, age, and city, race was not a significant predictor of advanced-stage prostate cancer. CONCLUSION: Low literacy may be an overlooked but significant barrier to the diagnosis of early-stage prostate cancer among low-income white and black men. The development of culturally sensitive, low-literacy educational materials may improve patient awareness of prostate cancer and improve the frequency of diagnosis of early-stage cancer.

Adult↗

Treating malignant pleural effusions cost consciously.

Malignant pleural effusions are associated with significant morbidity. Prompt clinical evaluation followed by aggressive treatment often results in successful palliation. This report summarizes the traditional and experimental approaches used in the management of malignant pleural effusion and provides an attempt at analysis of cost comparison and resource utilization associated with the use of various sclerosing agents. The standard sclerotherapy for malignant pleural effusions has routinely been performed as an inpatient procedure using a large-bore chest tube for drainage and instillation of a sclerosing agent. Use of a small-bore catheter for drainage and pleurodesis is associated with reduced patient discomfort and appears to be feasible and equally efficacious in the ambulatory setting. Results with the ambulatory procedure are preliminary but promising. Future comparisons with the traditional approach will allow therapy to be based not only on efficacy, but also on the use and expense of related resources.

Cost-Benefit Analysis↗

Cancer insurance policies in Japan and the United States.

Cancer care in the United States often results in financial hardship for patients and their families. Standard health insurance covers most medical costs, but nonmedical costs (such as lost wages, deductibles, copayments, and travel to and from caregivers) are paid out of pocket. Over the course of treatment, these costs can become substantial. Insurance companies have addressed the burden of these out-of-pocket costs by offering supplemental cancer insurance policies that, upon diagnosis of cancer, pay cash benefits for items that usually require out-of-pocket expenditures and are distinct from reimbursements made by traditional health insurance. Limitations associated with managed care have fostered increased consumer awareness and interest in the United States for cancer insurance and its ability to defray treatment expenditures that usually require out-of-pocket payments. Marketing campaigns are becoming more aggressive, and the number of cancer insurance policies sold has been steadily rising. While cancer insurance is only recently gaining popularity in the United States, it has been a successful product in Japan for over twenty years. In Japan, approximately one-quarter of the population own cancer insurance, and ten-year retention rates are estimated at 75%. As a result, individuals are afforded good access to nonmedical cancer services. Understanding the factors that led to the success of cancer insurance in Japan may assist policymakers in evaluating cancer insurance policies as they become more prevalent in the United States.

Aged↗

Quality of life in metastatic prostate cancer among men of lower socioeconomic status: feasibility and criterion related validity of 3 measures.

PURPOSE: Identification of metastatic disease progression is often difficult but important. Previous studies of quality of life in metastatic disease have been limited by the small number of respondents who were not white or of lower socioeconomic status. Quality of life assessment is generally done using self-administration techniques but this method is of limited usefulness for patients of low socioeconomic status, many of whom have limited reading abilities. We evaluated the feasibility and validity of interviewer administration of 3 quality of life instruments for patients of low socioeconomic status with metastatic prostate cancer. MATERIALS AND METHODS: We used instruments previously validated with self-administration methodology, including the European Organization for Research and Treatment of Cancer-Quality of Life Questionnaire-30, Functional Assessment of Cancer Therapy-General Scale and Quality of Life Index. Subjects were men with metastatic prostate cancer with stable (78) or progressive (32) disease at 4 Veterans Affairs medical centers and 1 other site. Of the patients 94% were Veterans Affairs patients and more than 60% were black. RESULTS: Each quality of life instrument required less than 10 minutes of interviewer administration and was able to discriminate between patients with stable versus progressive disease on several health status domains. CONCLUSIONS: These data support the feasibility and validity of quality of life measurement in patients of low socioeconomic status with metastatic prostate cancer. Consideration should be given to adding quality of life instruments to patient encounter even among low socioeconomic status, low literacy populations.

Aged↗

Predicting in-hospital mortality of patients with AIDS-related Pneumocystis carinii pneumonia: an example of hierarchically optimal classification tree analysis.

A non-linear statistical classification methodology known as hierarchically optimal classification tree analysis (CTA) offers promise of outperforming linear alternatives. We present the first example of CTA in medicine, for an application that uses four attributes (age, body mass index, alveolar-arterial oxygen gradient, prior history of AIDS) to predict in-hospital mortality for a sample of 1339 patients with AIDS-related Pneumocystis carinii pneumonia. We also illustrate use of a hold-out (cross-generalizability) sample that suggests a three-attribute model (alveolar-arterial oxygen gradient, total lymphocyte count, private insurance). Both CTA models achieved approximately 25 per cent of the total possible theoretical improvement beyond chance in classification accuracy (for comparative purposes, logistic regression and regression-tree models obtained in prior research returned less than 10 per cent). We illustrate how one can use CTA models to construct staging systems for assessing severity of illness, and for identifying important directions for future research. In the context of the example, we discuss additional advantages of CTA versus linear alternatives that include treatment of missing data, control of experimentwise type I error, richness of the substantive findings, and ease of use.

AIDS-Related Opportunistic Infections↗